In the 10 years that have passed since the Nobel prize-winning discovery of RNA
interference (RNAi), billions of dollars have been invested in the therapeutic application of …

Small interfering RNAs (siRNAs) are widely used to study gene function owing to the ease
with which they silence target genes, and there is considerable interest in their potential for …

One of the most significant challenges in the development of clinically viable delivery
systems for RNA interference therapeutics is to understand how molecular structures …

Small interfering RNAs (siRNAs) conjugated to a trivalent N-acetylgalactosamine (GalNAc)
ligand are being evaluated in investigational clinical studies for a variety of indications. The …

Dramatic advances in understanding of the roles RNA plays in normal health and disease
have greatly expanded over the past 10 years and have made it clear that scientists are only …

Abstract Changes in the patterns of gene expression are widely believed to underlie many of
the phenotypic differences within and between species. Although much emphasis has been …

RNA interference (RNAi) is a natural process through which expression of a targeted gene
can be knocked down with high specificity and selectivity. Using available technology and …

With increasing knowledge on the molecular mechanisms of endogenous RNA interference,
small interfering RNAs (siRNAs) have been emerging as innovative nucleic acid medicines …

Proprotein convertase subtilisin/kexin type 9 (PCSK9) regulates low density lipoprotein
receptor (LDLR) protein levels and function. Loss of PCSK9 increases LDLR levels in liver …

Transfection of small RNAs (such as small interfering RNAs (siRNAs) and microRNAs
(miRNAs)) into cells typically lowers expression of many genes. Unexpectedly, increased …